SBIR Phase I: Horizontal genomic transfer technology: Bioengineering DNA sequences for the creation of a non-viral targeted cancer cell specific gene therapy platform

The broader impact /commercial potential of this Small Business Innovation Research (SBIR) Phase I project is to improve clinical outcomes for cancer treatment. Horizontal genomic transfer (HGT) has the potential to deliver a variety of lethal payloads to tumors with high specificity. The proposed platform will enable better cancer therapies with lower side effects. To accelerate translation, this project proposes the development of a high-throughput platform to enable the evaluation of the specificity, lethality, and efficiency of therapies based on HGT across many different cell types, multiple dosing and many other variables. These defining features of the proposed HGT platform will permit the rapid and cost-effective development of novel gene therapies to improve patient care. This Small Business Innovation Research Phase I project aims to create a framework for the rapid high-throughput screening of cell-specific HGT-based therapeutics. HGT was previously thought to only be present in species like bacteria and fungi, but has recently been discovered in human cancer cells. Preliminary data has demonstrated the HGT’s abilities to deliver different expression vectors in animal models and in culture, but the efficiency of delivery and expression is currently unknown. This project will interrogate identified HGT and their sequences in the lab to better understand how to further optimize them to maximize delivery and expression of genomic payloads. The first aim proposes the development of the high-throughput screening platform for assessing integration and expression efficiency of HGT, and the second aim proposes the correlation of flow cytometry data with HGT integration into the tumor cell genome. Successful completion of the project will inform HGT platform development to evaluate numerous cellular parameters simultaneously and ultimately accelerate product development for cancer therapies and potentially in other indications. This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.